DT-216, Gene-TAC

Design Therapeutics is advancing a novel approach to activate transcription of the FXN gene and restore the production of frataxin protein. This approach is based on work from Dr. Aseem Ansari’s lab with the development of compounds called synthetic transcription elongation factors. In 2019, FARA awarded the Kyle Bryant Translational Research Award to Design Therapeutics for their research proposal “Advancing Syn-TEFs as a therapeutic candidate for F A”. This grant award allowed for some of the early tests in animal models. Design Therapeutics built on this early work to develop a next-generation molecule they call gene-targeting chimera or Gene-Tac.